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2021 1
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2023 37
2024 23

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Page 1
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study.
Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM. Neul JL, et al. Nat Med. 2023 Jun;29(6):1468-1475. doi: 10.1038/s41591-023-02398-1. Epub 2023 Jun 8. Nat Med. 2023. PMID: 37291210 Free PMC article. Clinical Trial.
Multidisciplinary Management of Rett Syndrome: Twenty Years' Experience.
Vilvarajan S, McDonald M, Douglas L, Newham J, Kirkland R, Tzannes G, Tay D, Christodoulou J, Thompson S, Ellaway C. Vilvarajan S, et al. Genes (Basel). 2023 Aug 11;14(8):1607. doi: 10.3390/genes14081607. Genes (Basel). 2023. PMID: 37628658 Free PMC article. Review.
Trofinetide: a pioneering treatment for Rett syndrome.
Parent H, Ferranti A, Niswender C. Parent H, et al. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/ Epub 2023 Jul 16. Trends Pharmacol Sci. 2023. PMID: 37460385
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Powers S, Likhite S, Gadalla KK, Miranda CJ, Huffenberger AJ, Dennys C, Foust KD, Morales P, Pierson CR, Rinaldi F, Perry S, Bolon B, Wein N, Cobb S, Kaspar BK, Meyer KC. Powers S, et al. Mol Ther. 2023 Sep 6;31(9):2767-2782. doi: 10.1016/j.ymthe.2023.07.013. Epub 2023 Jul 22. Mol Ther. 2023. PMID: 37481701 Free article.
Parental age effects and Rett syndrome.
Fang X, Baggett LM, Caylor RC, Percy AK, Neul JL, Lane JB, Glaze DG, Benke TA, Marsh ED, Motil KJ, Barrish JO, Annese FE, Skinner SA. Fang X, et al. Am J Med Genet A. 2024 Feb;194(2):160-173. doi: 10.1002/ajmg.a.63396. Epub 2023 Sep 28. Am J Med Genet A. 2024. PMID: 37768187
52 results